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PRAX-080

Development stage: Preclinical
Lead developer: Praxis Precision Medicines
Modality: Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
Administration: Intrathecal

Overview

PRAX-080 is an investigational antisense oligonucleotide (ASO) therapy developed to target PCDH19 mosaic expression, representing a novel approach for the treatment of PCDH19-related epilepsy. This rare genetic disorder is characterized by early-onset seizures and cognitive impairment, predominantly affecting females. The drug’s mechanism involves inhibiting or modulating the expression of the PCDH19 gene, which is implicated in this form of epilepsy. PRAX-080 is part of Praxis Precision Medicines’ pipeline focused on precision therapies for developmental and epileptic encephalopathies[2][3][4][5][7].

Targets

PCDH19

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